RARE Daily

Flagship Pioneering Enters First-of-its-Kind Partnership with Cystic Fibrosis Foundation to Develop CF Therapies

November 3, 2021

Venture capital firm Flagship Pioneering said it entered into a first-of-its-kind strategic partnership with the Cystic Fibrosis Foundation to develop therapeutics that address the unmet needs of people living with cystic fibrosis by leveraging the innovations and teams of multiple Flagship-founded companies.

Photo: Noubar Afeyan, Founder and CEO of Flagship Pioneering

Through this agreement, the Cystic Fibrosis Foundation has committed up to $110 million to Pioneering Medicines to bring forward multiple development candidates and develop these candidates to human proof of concept. Pioneering Medicines is a strategic initiative within Flagship Pioneering that is dedicated to conceiving and developing a broad portfolio of life-changing treatments by leveraging and expanding the use of Flagship’s innovations.

Pioneering Medicines and the Cystic Fibrosis Foundation will partner with multiple companies within the Flagship ecosystem, leveraging emerging technologies including Tessera Therapeutics’ Gene Writing technology, targeted delivery modalities, and RNA technologies. The teams will work together to harness their unique technologies and develop individual asset companies specifically focused on potential treatments for cystic fibrosis.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by mutations in the CFTR gene that lead to a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein. Children must inherit two defective CFTR genes—one from each parent—to have CF. There are approximately 2,000 known mutations in the CFTR gene. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

Despite recent advances, approximately 10 percent of individuals living with cystic fibrosis have rare or nonsense mutations and do not have any effective treatment options.

“This is a powerful opportunity to bring together the capabilities of multiple Flagship bioplatform companies, along with the disease expertise and extensive network of the Cystic Fibrosis Foundation, and the drug development capabilities of Pioneering Medicines to deliver innovative treatments for these patients and to explore the possibility of curative treatments for all those living with cystic fibrosis,” said Paul Biondi, president of Pioneering Medicines and executive partner of Flagship Pioneering. “This partnership highlights the benefits of the Pioneering Medicines model.”

Bondi said Pioneering Medicines is bringing together an integrated team to develop life-changing treatments and deliver benefits to patients much sooner than any company could independently deliver.

“There is a tremendous opportunity to expand the impact of Flagship’s innovative bioplatforms by creating medicines in disease areas beyond where the individual companies are initially focused,” said Noubar Afeyan, founder and CEO of Flagship Pioneering. “Through Pioneering Medicines and our new collaboration with the Cystic Fibrosis Foundation, we have an unparalleled opportunity to create innovative products that combine platform technologies to help address the unmet needs of the cystic fibrosis community.”

Author: Rare Daily Staff

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